In an engaging presentation, Skye C. Mayo, MD, MPH, shed light on both the potential and challenges of hepatic arterial infusion (HAI) pump chemotherapy in treating intrahepatic cholangiocarcinoma (ICC). As ICC cases rise globally, he emphasized the urgency of advancing liver-directed therapies that not only improve survival but also enhance the quality of life (QoL) for patients facing this aggressive disease.
Dr Mayo’s presentation opened with a discussion on the increasing incidence of ICC, particularly in underserved communities in Oregon. Some counties exhibit age-adjusted ICC rates significantly higher than the national average. Native American populations face a disproportionate burden, with liver and biliary tract cancer rates at least 3 times higher than the general population. Dr Mayo is actively collaborating with local health authorities and Tribal Nations to improve liver health and combat the substantial impact of ICC in these communities.
Despite advances in systemic therapies, the prognosis for ICC remains grim. Only 15% of ICC cases are resectable at diagnosis, and even after surgery, recurrence is common. Dr. Mayo argued that the term "disease recurrence" is a misnomer, as these cases often represent the progression of previously undetectable micro-metastatic disease. This underscores the limitations of current imaging technologies and the need for novel regional therapies like HAI to comprehensively address liver-confined disease.
HAI delivers chemotherapy directly to the liver via an implanted pump, allowing for high local drug concentrations with reduced systemic toxicity. Floxuridine is particularly effective in this setting due to its high first-pass metabolism in the liver. This approach is especially relevant for patients with multifocal or unresectable disease, where controlling tumor burden within the liver is critical. Clinical data from a phase 2 trial from Memorial Sloan Kettering Cancer Center (NCT01862315) reported a median overall survival of 25 months in patients with unresectable ICC treated with HAI plus systemic chemotherapy, longer than the 12 to 13 months observed with standard systemic regimens in earlier trials. In addition, a subset of patients became eligible for surgical resection, highlighting HAI’s ability to alter treatment trajectories.
Building on these results, the HELIX-1 (NCT04251715) trial is evaluating a strategy of induction FOLFIRINOX followed by HAI with floxuridine and dexamethasone in patients with more advanced disease. The trial expanded eligibility to include patients with multifocal disease, a group historically considered unsuitable for surgery. Laparoscopy revealed that 23% of patients initially thought to have a single lesion actually had multifocal disease.
Patients in the HELIX-1 trial had 100% disease control at 10 months and a median progression-free survival of 18 months. Two of the 5 patients transitioned to HAI chemotherapy for 6 months and later to no treatment at all for over a year.
Despite these outcomes, HAI therapy is complex and requires careful patient selection. Successful implementation depends on specialized multidisciplinary teams, and logistical factors, such as frequent visits and access to experienced centers, can limit its broader use. Access disparities also remain a concern, as relatively few centers currently offer this approach. Ongoing research is focused on optimizing this treatment and expanding its role through combination strategies. Early data suggest that pairing HAI with systemic treatments, including immunotherapy, may further enhance efficacy by targeting both local and systemic disease.
Importantly, individual patient experiences underscore the potential impact of this approach, with some having long-term disease control well beyond initial expectations. These outcomes highlight the evolving role of HAI as part of a broader effort to improve survival while maintaining QoL for patients with ICC.
Source: Mayo SC. Hepatic arterial infusion pump chemotherapy for intrahepatic cholangiocarcinoma: improving survival and QOL. Presented at: 2026 Annual Cholangiocarcinoma Foundation Conference. May 1-3, 2026; Salt Lake City, UT.
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